Myeloid MalignanciesPhase 2NCT06668584
A Phase II Open-label Study of Olutasidenib Post-transplant Maintenance Therapy for Patients With IDH1-mutated Myeloid Malignancies
A Phase 2 study testing Olutasidenib in people with Myeloid Malignancies, run by M.D. Anderson Cancer Center at 1 site in United States.
Trial summary
The goal of this clinical research study is to learn about the safety and tolerability of giving olutasidenib to patients with IDH1-mutated myeloid malignancies as maintenance therapy after they receive a stem cell transplant. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Myeloid Malignancies
- What is being tested?
- Olutasidenib
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 31 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Dec 2024to 31 Dec 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosis of AML, MDS, MPN, or CMML according to World Health Organization (WHO) classification that underwent first or second alloSCT regardless of stem cell source, donor type/match, conditioning regimen, or GVHD prophylaxis and is at least 30 days post stem cell transplant until day 120.
- Evdence of an IDH1 mutation presence by next generation sequencing panel at original diagnosis.
- Evidence of engraftment by Absolute neutrophil count (ANC) >/= 1.0x 109/L without daily use of myeloid growth factor (G-CSF) for at least 7 days; and Platelet >/= 30 x 109/L with out platelet transfusion within 1 week.
- Patient in morphologic remission with Day +30 bone marrow <5% blast. Will include MRD positive or negative.
- Age 18 to 75 years old.
- ECOG performance status of 0, 1, 2. Or KPS above 70.
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- Creatinine clearance greater or equal to 40cc/min as defined by the Cockcroft-Gault Equation.
- Males (mL/min): (140-age) IBW (kg) / 72(serum creatinine(mg/dl)) Females (mL/min): 0.85(140-age) IBW (kg) / 72(serum creatinine(mg/dl)).
- Serum bilirubin </= 1.5 x upper limit of normal (ULN) except in subjects with Gilbert's Syndrome in whom total bilirubin must be </= 3.0 mg/dl. Aspartate transaminase (AST) or alanine transaminase (ALT) </= 2.5 x ULN. Alkaline phoshatase </=2.5 x ULN.
- No active bleeding.
- No clinical evidence of life-threatening infection.
- Capable of understanding the investigational nature, potential risks, and benefits of the study, and able to provide valid informed consent.
- Negative serum or urine pregancy test for wome with reproductive potential at screening.
- Female participants of non-childbearing potential must meet at least one of the following criteria:
- Postmenopausal (no menses in greater than or equal to 12 consecutive months).
- History of hysterectomy or bilateral salpingo-oophorectomey.
- Ovarian failure (follicle-stimulating hormone and Estradiolin menopausal range, who have received Whole Pelvic Radiation Therapy).
- History of bilateral tubal ligation or another surgical sterilization procedure
- Subjects who are of childbearing potential, sexually active, and at risk of pregnancy must agree to use a highly effective method of contraception for the duration of the active treatment and at least 3 months post-completion of the study therapy. See Appendix B.
- Approved methods of birth control are as follows: Hormonal contraception (i.e., birth control pills, injection, implant, transdermal patch, vaginal ring), Intrauterine device (IUD), Tubal Ligation or hysterectomy, Subject/Partner post vasectomy, Implantable or injectable contraceptives, and condoms plus spermicide. Not engaging in sexual activity for the total duration of the trial and the drug washout period is an acceptable practice however periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of birth control. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. See Appendix B.
- Men treated or enrolled in this protocol must also agree to use adequate contraception prior to the study, for the duration of study participation, and 4 months after completion of Olutasidenib administration.
Who cannot join
- Use of any of the following after transplantation and prior to starting study therapy. Anti-leukemic agents given as post-transplant maintenance therapy (e.g., subcutaneous, or oral 5-azacitidine or FL T3 inhibitors for maintenance)
- Overall grade II-IV aGVHD. However, upon complete resolution of aGVHD-related symptoms with grade 1 or 0 overall clinical grade would be appropriate to enroll at that time. Patients may be eligible for enrollment if they are on prednisone 0.5 mg/kg daily dose or lower, tacrolimus, sirolimus, and/or ruxolitinib.
- cGVHD, moderate or servere by NIH criteria.
- Active uncontrolled systemic fungal, bacterial, or viral infection. However, patients receiving anti-micobial agents including antibiotics, antiviral and antifungal therapies are allowed if the infection is controlled, and the patient is hemodynamically stable.
- Known active viral infection with Human Immunodeficiency Virus (HIV), Hepatitis B Virus (HBV), or Hepatitis C Virus (HCV).
- Patients with known active hepatitis B virus (HBV) infection will be excluded because of potential effects on immune function and/or drug interactions. However, if a patient has HBV history with an undetectable HBV load by polymerase chain reaction (PCR), no liver-related complications, and is on definitive HBV therapy that is not contraindicated in this study, then they would be eligible for study.
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- Patients with known active hepatitis C virus (HCV) infection will be excluded because of potential effects on immune function and/or drug interactions. However, if a patient with a history of HCV infection has received definitive therapy (and is now HCV viral load negative), or if a patient has a reactive HCV antibody test but has an undetectable viral load by PCR, then they would be eligible.
- Patients with known active HIV infection will be excluded out of concern for the drug-drug interaction with venetoclax and highly active antiretroviral therapy (HART)
- QT prolongation of corrected QTcF interval (QTc) > 480 milliseconds
- Any other condition that would, in the Investigator's judgment, contraindicate the patient's participation in the clinical study due to safety concerns with clinical study procedures.
- Patients with cognitive impairments or psychiatric disorders that can interfere with safety or with obtaining informed consent of compliance with study procedures.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
The University of Texas M. D. Anderson Cancer Center
Houston, Texas, 77030
Jeremy Ramdial, MD, 713-745-0146, jlramdial@mdanderson.org
Jeremy Ramdial, MD
About this study
The goal of this clinical research study is to learn about the safety and tolerability of giving olutasidenib to patients with IDH1-mutated myeloid malignancies as maintenance therapy after they receive a stem cell transplant.
Read the detailed description
Primary Objective:
To determine the safety and tolerability of olutasidenib as maintenance post-alloSCT.
Secondary Objectives:
- To determine the rate of progression-free survival (PFS) of patients with IDH1 mutated myeloid malignancies who have undergone a stem cell transplant on olutasidenib post-transplant.
II. To determine response rate, overall survival (OS), cumulative incidence of relapse, non-relapse mortality (NRM), graft versus host disease (GVHD) relapse-free survival (GRFS), rate and grading of acute GVHD (aGVHD) grade 2-4 and 3-4 at day 100, incidence and grading chronic GVHD (cGVHD) all grades.
OUTLINE:
Starting 30-120 days post-stem cell infusion, patients receive olutasidenib orally (PO) twice daily (BID) on days 1-28 of each cycle. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity. Patients also undergo blood sample collection throughout the study. Additionally, patients may undergo bone marrow aspiration and biopsy, echocardiography (ECHO)/multigated acquisition (MUGA) scan and chest x-ray at screening.
- Conditions
- Myeloid Malignancies
- Being tested
- Olutasidenib
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OlutasidenibDrug
Given by mouth
| Study group | Role | What they receive |
|---|---|---|
| Olutasidenib Maintenance Therapy | Experimental | Starting 30-120 days post-stem cell infusion, patients receive olutasidenib PO BID on days 1-28 of each cycle. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity. Patients also undergo blood sample collection throughout the study. Additionally, patients may undergo bone marrow aspiration and biopsy, ECHO/MUGA scan and chest x-ray at screening. Receives: Drug: Olutasidenib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Adverse Events (AEs)
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Measured: Through study completion; an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Oct 2024
- Study start31 Dec 2024
- Primary completion31 Dec 2027 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myeloid MalignanciesStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 31 participants (estimated)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jeremy Ramdial, MD, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06668584
- Sponsor's protocol ID
- 2024-0522
- Other IDs
- NCI-2025-00104
- Status last verified
- Sep 2026
- First submitted
- 30 Oct 2024
- Last updated
- 5 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Open-label Study of Olutasidenib Post-transplant Maintenance Therapy for Patients With IDH1-mutated Myeloid Malignancies
- ClinicalTrials.gov ID
- NCT06668584
- Acronym / sponsor ID
- 2024-0522
- Conditions
- Myeloid Malignancies
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 31 participants (estimated)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 31 Dec 2024 (actual)
- Primary completion
- 31 Dec 2027 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 31 Oct 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this clinical research study is to learn about the safety and tolerability of giving olutasidenib to patients with IDH1-mutated myeloid malignancies as maintenance therapy after they receive a stem cell transplant.
Detailed description
Primary Objective:
To determine the safety and tolerability of olutasidenib as maintenance post-alloSCT.
Secondary Objectives:
- To determine the rate of progression-free survival (PFS) of patients with IDH1 mutated myeloid malignancies who have undergone a stem cell transplant on olutasidenib post-transplant.
II. To determine response rate, overall survival (OS), cumulative incidence of relapse, non-relapse mortality (NRM), graft versus host disease (GVHD) relapse-free survival (GRFS), rate and grading of acute GVHD (aGVHD) grade 2-4 and 3-4 at day 100, incidence and grading chronic GVHD (cGVHD) all grades.
OUTLINE:
Starting 30-120 days post-stem cell infusion, patients receive olutasidenib orally (PO) twice daily (BID) on days 1-28 of each cycle. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity. Patients also undergo blood sample collection throughout the study. Additionally, patients may undergo bone marrow aspiration and biopsy, echocardiography (ECHO)/multigated acquisition (MUGA) scan and chest x-ray at screening.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Olutasidenib Maintenance Therapy | Experimental | Drug: OlutasidenibStarting 30-120 days post-stem cell infusion, patients receive olutasidenib PO BID on days 1-28 of each cycle. Cycles repeat every 28 days for up to 2 years in the absence of disease progression or unacceptable toxicity. Patients also undergo blood sample collection throughout the study. Additionally, patients may undergo bone marrow aspiration and biopsy, ECHO/MUGA scan and chest x-ray at screening. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (21 listed)
- Diagnosis of AML, MDS, MPN, or CMML according to World Health Organization (WHO) classification that underwent first or second alloSCT regardless of stem cell source, donor type/match, conditioning regimen, or GVHD prophylaxis and is at least 30 days post stem cell transplant until day 120.
- Evdence of an IDH1 mutation presence by next generation sequencing panel at original diagnosis.
- Evidence of engraftment by Absolute neutrophil count (ANC) >/= 1.0x 109/L without daily use of myeloid growth factor (G-CSF) for at least 7 days; and Platelet >/= 30 x 109/L with out platelet transfusion within 1 week.
- Patient in morphologic remission with Day +30 bone marrow <5% blast. Will include MRD positive or negative.
- Age 18 to 75 years old.
Key exclusion criteria (11 listed)
- Use of any of the following after transplantation and prior to starting study therapy. Anti-leukemic agents given as post-transplant maintenance therapy (e.g., subcutaneous, or oral 5-azacitidine or FL T3 inhibitors for maintenance)
- Overall grade II-IV aGVHD. However, upon complete resolution of aGVHD-related symptoms with grade 1 or 0 overall clinical grade would be appropriate to enroll at that time. Patients may be eligible for enrollment if they are on prednisone 0.5 mg/kg daily dose or lower, tacrolimus, sirolimus, and/or ruxolitinib.
- cGVHD, moderate or servere by NIH criteria.
- Active uncontrolled systemic fungal, bacterial, or viral infection. However, patients receiving anti-micobial agents including antibiotics, antiviral and antifungal therapies are allowed if the infection is controlled, and the patient is hemodynamically stable.
- Known active viral infection with Human Immunodeficiency Virus (HIV), Hepatitis B Virus (HBV), or Hepatitis C Virus (HCV).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OlutasidenibGiven by mouth | Drug | Olutasidenib Maintenance Therapy |
Primary outcome
Safety and Adverse Events (AEs)
Time frame: Through study completion; an average of 1 year
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Oct 2026.
- MD Anderson Cancer Center WebsiteLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jeremy Ramdial, MD principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Olutasidenib in people with Myeloid Malignancies, run by M.D. Anderson Cancer Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 21 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jeremy Ramdial, MD ((713) 745-0146, jlramdial@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety and Adverse Events (AEs) (time frame: Through study completion; an average of 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Oct 2026.